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Results for “Inherited Metabolic Disorder of Nervous System”

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Showing 20 of 0 results

Testing effectiveness (Phase 2)Looking for participantsNCT06634108
What this trial is testing

Genistein in trAnSthyretin recePtor Amyloid caRdiomyopathy

Who this might be right for
Amyloid Cardiomyopathy
London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's 20
Testing effectiveness (Phase 2)Study completedNCT00446550
What this trial is testing

Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease

Who this might be right for
Gaucher DiseaseType 1 Gaucher DiseaseGaucher Disease, Type 1
Amicus Therapeutics 19
Not applicableStudy completedNCT04141891
What this trial is testing

Advancing Understanding of Transportation Options

Who this might be right for
Diabetic RetinopathyMacular DegenerationGlaucoma+29 more
University of Colorado, Denver 529
Not applicableLooking for participantsNCT01780168
What this trial is testing

The NIH MINI Study: Metabolism, Infection, and Immunity in Inborn Errors of Metabolism

Who this might be right for
Oxidative Phosphorylation DeficienciesElectron Transport Chain Disorders, MitochondrialMitochondrial Disorders+1 more
National Human Genome Research Institute (NHGRI) 500
Not applicableNot Yet RecruitingNCT07173010
What this trial is testing

Pediatric Arthropathy Beyond Inflammation: Clinical Spectrum and Diagnostic Approach at Assiut University Children Hospital

Who this might be right for
MucopolysaccharidosesProgressive Pseudorheumatoid DysplasiaFarber Disease+3 more
Assiut University 35
Large-scale testing (Phase 3)Active Not RecruitingNCT05371613
What this trial is testing

Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)

Who this might be right for
Mucopolysaccharidosis II
Denali Therapeutics Inc. 65
Testing effectiveness (Phase 2)Study completedNCT01855360
What this trial is testing

Doxycycline and TUDCA in Patients With Transthyretin Amyloid Cardiomyopathy

Who this might be right for
Amyloidosis; Heart (Manifestation)Senile Cardiac Amyloidosis
Brigham and Women's Hospital 40
Not applicableStudy completedNCT00001289
What this trial is testing

Effects of Enzyme Replacement in Gaucher's Disease

Who this might be right for
Gaucher's Disease
National Institute of Neurological Disorders and Stroke (NINDS) 70
Large-scale testing (Phase 3)Study completedNCT01614574
What this trial is testing

Study of Velaglucerase Alfa Enzyme Replacement Therapy in Japanese Patients With Gaucher Disease

Who this might be right for
Gaucher Disease
Shire 6
Not applicableStudy completedNCT04470713
What this trial is testing

Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2

Who this might be right for
GM1 GangliosidosisGM2 GangliosidosisGaucher Disease, Type 2+3 more
Idorsia Pharmaceuticals Ltd. 226
Large-scale testing (Phase 3)Looking for participantsNCT06628128
What this trial is testing

A Long-Term Study of JNT-517 in Participants With Phenylketonuria

Who this might be right for
Phenylketonuria (PKU)
Otsuka Pharmaceutical Development & Commercialization, Inc. 240
Not applicableActive Not RecruitingNCT05526664
What this trial is testing

Omics Gaucher Study: Multiomic Approach

Who this might be right for
Gaucher Disease
CENTOGENE GmbH Rostock 25
Large-scale testing (Phase 3)Study completedNCT02277431
What this trial is testing

Probiotic Supplementation Among Adults With Recurrent Gastrointestinal Symptoms

Who this might be right for
Gastrointestinal SymptomsBloatingGas+3 more
University of Maryland, Baltimore 80
Post-approval studies (Phase 4)Study completedNCT04656600
What this trial is testing

Study to Evaluate Efficacy and Safety of Imiglucerase Treatment in Chinese Patients With Gaucher Disease Type Ⅲ

Who this might be right for
Gaucher's Disease
Sanofi 12
Post-approval studies (Phase 4)Study completedNCT06019728
What this trial is testing

A Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme

Who this might be right for
Fabry's Disease
Sanofi 8
Testing effectiveness (Phase 2)Study completedNCT04817111
What this trial is testing

NAD+ Precursor Supplementation in Friedreich's Ataxia

Who this might be right for
Friedreich Ataxia
Metro International Biotech, LLC 7
Testing effectiveness (Phase 2)Study completedNCT01885936
What this trial is testing

Safety and Efficacy of Albuterol in Individuals With Late-onset Pompe Disease

Who this might be right for
Pompe Disease
Duke University 16
Not applicableStudy completedNCT02403687
What this trial is testing

Prospective Analgesic Compound Efficacy (PACE) Study

Who this might be right for
ArthritisTendonitisGout+5 more
Express Specialty Pharmacy 300
Testing effectiveness (Phase 2)Study completedNCT02004704
What this trial is testing

A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency

Who this might be right for
Sphingomyelin Lipidosis
Genzyme, a Sanofi Company 25
Not applicableStudy completedNCT06448871
What this trial is testing

Ultrasound to Assess Sarcopenia in Prader Willi Syndrome

Who this might be right for
Prader-Willi SyndromeSarcopeniaMetabolic Syndrome
Taipei Tzu Chi Hospital, Buddhist Tzu Chi Medical Foundation 49
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